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JCI early table of contents for July 1, 2013

JCI Journals

Potential gene therapy for Sickle cell disease

Sickle cell disease (SCD) is an autosomal recessive disorder caused by mutations in hemoglobin (HBB) that deform red blood cells. A small number of patients have been successfully treated with allogeneic hematopoietic stem cell (HSC) transplantation; however, there are several drawbacks and complications associated with this procedure, including graft vs. host disease and long-term immune suppression. Many of complications could potentially be avoided by the use of autologous HSC transplant, which uses patient cells that have been treated genetically modified to replace defective hemoglobin. In this issue of the Journal of Clinical Investigation , Zulema Romero and colleagues at UCLA investigated the utility of a genetic vector encoding a human hemoglobin gene engineered to impede sickle hemoglobin and prevent the sickling of red blood cells. The vector was used to efficiently transduce bone marrow cells from SCD patients and the transduced cells were successfully transplanted into immunocompromised mice, suggesting that this method could potentially be used to treat SCD.

TITLE: β-globin gene transfer in human bone marrow for sickle cell disease

University of California, Los Angeles (UCLA), Los Angeles, CA, USA
Phone: 1-310-794-1884; Fax: 310-267-2774; E-mail:

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Cross-species malaria immunity induced by chemically attenuated parasites

Malaria, a mosquito-born infectious disease, kills over 600,000 people every year. Research has focused on the development of a vaccine to prevent the disease; however, many malaria vaccines targeting parasite antigens have failed because the antigen targets are highly variable. Based on the observation that low-density infections can induce antibody-independent immunity to different malaria strains, Michael Good and colleagues at Griffith University in Australia created a vaccine using blood-stage malaria parasites that were attenuated with a chemical agent that keeps the parasite from multiplying. In this issue of the Journal of Clinical Investigation, they demonstrate that mice inoculated with a single species of attenuated parasite display immunity to multiple malaria species for over 100 days. These data indicate that vaccination with chemically attenuated parasites provides protective immunity and suggest that such vaccines could be used to target human malaria species.

TITLE: Cross-species malaria immunity induced by chemically attenuated parasites

AUTHOR CONTACT: Michael Batzloff
Griffith University, Gold Coast, AUS
Phone: 5552 9434; E-mail:

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A potential gene therapy for Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis Type IIIA (MPSIIIA) is a metabolic disorder in which the body is missing an enzyme that is required to break down long chains of sugars known as glycosaminoglycans. Over time, the glycosaminoglycans collect in the body and cause damage, particularly in the brain. In this issue of the Journal of Clinical Investigation, Fàtima Bosch and colleagues at Universitat Autònoma de Barcelona in Spain developed a form of gene therapy to replace the enzyme that is missing in MPSIIIA. By injecting the replacement gene into the the cerebrospinal fluid that surrounds the brain and spinal cord, Bosch and colleagues found that they could successfully deliver a replacement gene to the brain in mice and dogs. This study demonstrates that gene therapy can be delivered to the brain through the cerebrospinal fluid and suggests that this approach could potentially be used as a therapy for MPSIIIA.

TITLE: Whole body correction of Mucopolysaccharidosis IIIA by intra-cerebrospinal fluid gene therapy

Universitat Autònoma de Barcelona, Bellaterra, UNK, ESP
Phone: 0034935814182; Fax: 0034935814180; E-mail:

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TITLE: Developmental differences in IFN signaling affect GATA-1s induced megakaryocyte hyperproliferation

Children's Hospital Boston/Harvard Medical School, Boston, MA, USA
Phone: 617-919-2026; Fax: 617-730-0222; E-mail:

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TITLE: Inactivation of specific beta-cell transcription factors in Type 2 diabetes

Vanderbilt University Medical Center, Nashville, TN, USA
Phone: 615-322-7027; Fax: 615-322-7236; E-mail:

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TITLE: Colon cancer progression is driven by APEX1-mediated up-regulation of Jagged

Chosun University School of Medicine, Gwangju, KOR
Phone: 82-62-230-6337; Fax: 82-62-230-6337; E-mail:

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TITLE: Angiopoietin 2 mediates microvascular and hemodynamic alterations in sepsis

AUTHOR CONTACT: Christian Kupatt
Klinikum Grosshadern, Ludwig-Maximilians-University, Munich, DEU
Phone: +49-89-7095-6092; Fax: +49-89-7095-6075; E-mail:

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TITLE: Neutrophil extracellular traps sequester circulating tumor cells and promote metastasis

MUHC, Montreal, PQ, CAN
Phone: 514-934-1934 ext. 44327; Fax: 514-398-6769; E-mail:

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TITLE: Lipocalin 2 deactivates macrophages and worsens pneumococcal pneumonia outcomes

Medical University Vienna, Vienna, AUT

Phone: +43-1-40400-5139; Fax: +43-1-40400-5167; E-mail:

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TITLE: Gray platelet syndrome and defrective thrombo-inflammation in Nbeal2-deficient mice

AUTHOR CONTACT: Bernhard Nieswandt
University Hospital Würzburg and Rudolf Virchow Center, Würzburg, , DEU
Phone: +49 931 31 80405; Fax: +49 931 201 48123; E-mail:

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